Trials / Active Not Recruiting
Active Not RecruitingNCT04293185
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the BB305 Lentiviral Vector in Subjects With Sickle Cell Disease
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 35 (estimated)
- Sponsor
- Genetix Biotherapeutics Inc. · Industry
- Sex
- All
- Age
- 2 Years – 50 Years
- Healthy volunteers
- Not accepted
Summary
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | bb1111 | Drug Product is administered by IV infusion following myeloablative conditioning with busulfan. |
Timeline
- Start date
- 2020-02-14
- Primary completion
- 2027-05-01
- Completion
- 2027-11-01
- First posted
- 2020-03-03
- Last updated
- 2024-12-10
Locations
9 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04293185. Inclusion in this directory is not an endorsement.